The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to psilocybin-assisted therapy from Compass Pathways Ltd. (London) for patients with treatment-resistant depression (TRD), the manufacturer reports.

This lays the groundwork for an upcoming multi-country study that will assess the safety and efficacy of psilocybin, the psychoactive ingredient in psychedelic or “magic” mushrooms.

Robin Carhart-Harris, PhD, head of the psychedelic research group, Imperial College London, United Kingdom, noted in a press release that he and a group of investigators “found promising signals of efficacy and safety as treatment” for refractory depression in their 2015 study.

The new Breakthrough Therapy designation “is a strong endorsement for the potential of psilocybin therapy. We look forward to learning more as further clinical trials are carried out, by our team at Imperial College as well as in Compass’s multi-center trial,” Carhart-Harris said.

Previous research has shown benefit from psilocybin in treating end-of-life anxiety and depression, alcohol and tobacco addiction, and even obsessive-compulsive disorder.

As reported by Medscape Medical News, investigators from Imperial College assessed pre- and post-treatment MRI data for 19 patients with TRD who received psilocybin in a study published October 2017 in Scientific Reports. The patients experienced significant improvements in depressive symptoms after just 1-day following treatment, and almost half met criteria for a full response at 5 weeks. In addition, whole-brain analyses showed decreases in cerebral blood flow in the temporal cortex and increased resting-state functional connectivity after treatment.

“During exposure to psilocybin, the brain is in a different, more fluid state, where it is not possible to continue having their traditional negative thinking patterns, and in the majority, the brain resets into a nondepressive mode,” lead author David Nutt, MD, PhD, professor of neuropsychopharmacology at Imperial College, said.

Also commenting on the study, Frederick Barrett, PhD, Behavioral Pharmacology Research Unit, Johns Hopkins School of Medicine, called the findings “groundbreaking.”

Barrett added that the study “begins to put some dimensions to the potential neurobiological basis of the therapeutic effects of psychedelics.”

However, he cautioned that more research was needed before clinicians should move forward in treating TRD with the drug.

The FDA designates a drug as a Breakthrough Therapy “if preliminary clinical evidence shows that it may demonstrate substantial improvement over available therapy,” Compass noted in its release. It also highlights “the importance of supporting early research that can be translated to clinically meaningful outcomes.”

Overall, the designation “is a significant milestone for psilocybin therapy and a testament to work done over many years by research teams in the US, the UK, and Switzerland,” the company added. It reported that it is now running the first large-scale clinical trial for treating TRD with the drug. The study will be conducted in Europe and North America and will run for approximately 1 year.

“This is great news for patients,” George Goldsmith, executive chairman at Compass Pathways, added in the same release. “The FDA will be working closely with us to expedite the development process and increase the chances of getting this treatment to people suffering with depression as quickly as possible,” Goldsmith reported.

Steve’s Take:

Talk about an irresistible force. Seldom have I seen such rapid momentum for the FDA’s approval as this naturally occurring psychedelic compound called psilocybin. It was only two weeks ago that I wrote in a piece that Johns Hopkins researchers, among others, were encouraging the FDA to approve “Shrooms” for treatment-resistant depression.

And Breakthrough Therapy designation will supercharge the pace at which progress might occur in bringing long-needed treatment to the millions of patients around the globe struggling with this terrible mental illness.

For years, many scientists have been theorizing that hallucinating on so-called magic mushrooms (also called Little Smoke and Purple Passion) could possibly reboot the brain and clear out negative thoughts that may contribute to depression.

Regulatory approvals for the trial have already been granted in the UK, the Netherlands and Canada. The latest, FDA-blessed trial is expected to be a Phase 2b dose-ranging study involving 216 patients in 12 to 15 research sites across Europe and North America. The tests will begin in the UK soon and sites in other countries will get underway as soon as additional regulatory approvals are received. If the trial is successful, the company said it will be followed by Phase 3 studies.

“Depression is the leading cause of ill-health and disability worldwide, and treatment-resistant depression affects more than 100 million people. It is a huge unmet need and the trial will teach us more about how this new approach might address it,” Compass CEO George Goldsmith said in a statement.

Ekaterina Malievskaia, chief medical officer and co-founder of the company, emphasized that the study has been a collaborative effort with scientists, clinicians, patient representatives and regulators from all over the world.

Bottom Line:

This UK-based company is on the threshold of a financial bonanza given the stupendous size of the patient market. Perhaps not as large as cannabidiol, the naturally occurring cannabinoid constituent of cannabis, but massive.

High-profile Silicon Valley billionaire Peter Thiel, who invested an undisclosed amount in 2016, was one of Compass’s first high-profile backers. Other founding investors include Apeiron Investment Group and Galaxy Investment Partners.

If and when promising Phase 2 data start to emerge from the clinic, buckle your seat belts; Compass is sure to rocket skyward. If all goes as planned, stay tuned for what promises to be one of the hottest IPOs ever in the healthcare sector.